Mt. Sinai researchers are testing a powerful gene therapy, delivered directly into the heart, that has been shown in preliminary studies to reverse heart failure.
The preclinical findings, reported by heart specialists at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai, have shown that the so-called SUMO-1 gene therapy shrinks an enlarged heart, improves heart function, and increases blood flow.
"SUMO-1 gene therapy may be one of the first treatments that can actually shrink enlarged hearts and significantly improve a damaged heart's life-sustaining function," said Roger J. Hajjar, M.D., who led the study, published in the journal
Science Translational Medicine.
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"We are very eager to test this gene therapy in our patients suffering from severe heart failure."
Dr. Jajjar said the researchers now hope to begin human clinical trials of SUMO-1, which is named after a gene that is "missing in action" in heart failure patients. It is the second potential gene therapy technique developed by Dr. Jajjar's team.
Heart failure is a leading cause of hospitalization in the elderly, accounting for about 300,000 deaths each year in the United States. It occurs when a person's heart is too weak to pump and circulate blood.
The study was funded, in part, by the National Institutes of Health.
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